Mini-guts aid cystic fibrosis therapy

A new way to find the optimal cocktail of drugs for patients with cystic fibrosis has been announced by Dutch researchers.
24 June 2016


A new way to find the optimal cocktail of drugs for patients with cystic fibrosis has been announced by Dutch researchers.

Carried by up to one adult in every 25, cystic fibrosis is one of the most common inherited diseases. Sufferers of the condition, who inherit a faulty copy of the cystic fibrosis gene from both of their parents, produce thicker-than-normal mucus in their lungs, intestines and other sites.

Cystic fibrosis causes a range of health problems including an impaired ability to absorb sufficient calories and repeated chest infections.

Although the condition cannot be cured, as yet, drugs now entering the clinic can be used to "repair" and restore some of the functions of the faulty protein made by the cystic fibrosis gene. But because there are more than 2000 genetic changes capable of producing cystic fibrosis, and not all of them will respond equivalently to the available drugs, identifying the best therapy for a patient is currently a slow and hit-and-miss affair.

Now, writing in Science Translational Medicine, researcher Jeffery Beekman, from the Wilhelmina Children's Hospital in Utrecht, Holland, has pioneered a system he dubs the "mini-gut" to screen rapidly for agents capable of producing the best clinical results in patients.

Beekman's approach is to take a small biopsy from the patient's intestine, extract the stem cells and use these to grow small blobs of tissue.

"These are the mini-guts, or you could even say mini-bums, because we get the cells from the rectum," remarks Beekman wryly.

While they don't grossly resemble an intestine, or even a bottom, these mini-guts, which take about 3 weeks to grow, do give a very clear picture of how that patient's tissues respond to various therapies.

"So we can try a range of drugs to identify what will and won't work very rapidly," points out Beekman. "We can also freeze them. So a patient gives a sample once, we make the mini-guts and then put them in the freezer. Whenever we want to test a new therapy, we can thaw out one of the frozen samples and try it, without bothering the patient."


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